Lichen PlanusPhase 2NCT01041625
Pilot Study of Apremilast (CC-10004) in the Treatment of Moderate to Severe Lichen Planus
A Phase 2 study testing Apremilast (CC-10004) in people with Lichen Planus, run by Virginia Clinical Research, Inc. at 1 site in United States.
Trial summary
This study is designed to demonstrate to efficacy and safety of Apremilast 20mg oral administration twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis is that the subjects will achieve a significant clinical improvement in their skin disease according to a specialized physician… Read more about this study
- Who is it for?
- Ages 18 years and olderHealthy volunteers can take part
- Conditions the study is for
- Lichen Planus
- What is being tested?
- Apremilast (CC-10004)
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- Feb 2010to Feb 2012
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Must understand and voluntarily sign an informed consent form
- Must be male or female and aged ≥ 18 years at time of consent
- Must be able to adhere to the study visit schedule and other protocol requirements
- Subjects must have stable cutaneous lichen planus appropriate for systemic therapy based on the following criteria:
- Rated PGA of ≥ 3 (moderate or severe) AND
- ≥ 20 distinct lesions of lichen planus OR
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- Refractory to topical corticosteroid therapy (at least 4 weeks of high potency corticosteroid without significant improvement) OR
- Severe itching/pain that significantly impairs activities of daily living (i.e. working, school, sleep, etc.)
- Subjects using topical corticosteroids must be tapered and must undergo a washout period prior to initiation of the study. The washout period for topical corticosteroids is 2 weeks.
- Must meet the following laboratory criteria:
- Hemoglobin > 12 g/dL
- White blood cell (WBC) count ≥ 3000 /μL (≥ 3.0 X 109/L) and ≤ 14,000/μL (< 14 X 109/L)
- Platelets ≥ 100,000 /μL (≥ 100 X 109/L)
- Serum creatinine ≤ 1.5 mg/dL (or ≤ 133 μmol/L)
- Total bilirubin ≤ 2.0 mg/dL
- Aspartate transaminase (AST [serum glutamic oxaloacetic transaminase, SGOT]) and alanine transaminase (ALT [serum glutamate pyruvic transaminase, SGPT]) ≤ 1.5x upper limit of normal (ULN)
- Females of childbearing potential (FCBP) must have a negative urine pregnancy test at screening (Visit 1). In addition, sexually active FCBP must agree to use TWO of the following adequate forms of contraception while on study medication: oral, injectable, or implantable hormonal contraceptives; tubal ligation; intrauterine device; barrier contraceptive with spermicide; or vasectomized partner while on study. A FCBP must agree to have pregnancy tests every 4 weeks while on study.
- Males (including those who have had a vasectomy) must agree to use barrier contraception (latex condoms) when engaging in sexual activity with FCBP while on study medication and for 84 days after taking the last dose of study medication
Who cannot join
- Inability to provide voluntary consent
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Pregnant or breastfeeding
- Systemic fungal infection
- History of active mycobacterial infection with any species (including Mycobacterium tuberculosis) within 3 years prior to screening visit. Subjects with Mycobacterium tuberculosis infection more than 3 years prior to screening visit are allowed if successful treatment was completed at least 3 years prior to randomization and is documented and available for verification.
- Latent Mycobacterium tuberculosis infection as indicated by a positive Purified Protein Derivative [PPD] skin test. Subjects with a positive PPD skin test and documented completion of treatment for latent TB are eligible. Subjects with a positive PPD skin test and not treated or no documentation of completion of treatment are ineligible.
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- If QuantiFERON® test is performed instead of the PPD test, only those with a negative QuantiFERON® test are allowed in the study.
- History of incompletely treated Mycobacterium tuberculosis infection as indicated by
- Subject's medical records documenting incomplete treatment for Mycobacterium tuberculosis
- Subject's self-reported history of incomplete treatment for Mycobacterium tuberculosis
- History of recurrent bacterial infection (at least 3 major infections resulting in hospitalization and/or requiring intravenous antibiotic treatment within the past 2 years)
- Clinically significant abnormality on the chest x-ray (CXR) at screening. Chest x-rays performed within 3 months prior to start of study drug are acceptable.
- Use of topical cyclosporine, tacrolimus, or pimecrolimus within 2 weeks prior to start of study drug
- Use of systemic or intralesional corticosteroids, systemic retinoids, antimalarials, azathioprine, methotrexate, mycophenolate mofetil, dapsone, thalidomide, sulfasalazine, cyclosporine, metronidazole, griseofulvin, or phototherapy within 4 weeks prior to start of study drug
- Use of etanercept within 8 weeks prior to start of study drug.
- Use of adalimumab or infliximab within 12 weeks prior to start of study drug
- Use of alefacept within 24 weeks prior to start of study drug.
- Use of any investigational medication within 4 weeks prior to start of study drug or 5 pharmacokinetic/pharmacodynamic half-lives (whichever is longer)
- Any clinically significant abnormality on 12-lead ECG at screening
- History of congenital or acquired immunodeficiency (eg, Common Variable Immunodeficiency [CVID])
- Hepatitis B surface antigen positive or Hepatitis B core antibody positive at screening
- History of Human Immunodeficiency Virus (HIV) infection
- Antibodies to Hepatitis C at screening
- Positive ANA at screening visit
- Malignancy or history of malignancy (except for treated [ie, cured] basal- cell skin carcinomas > 3 years prior to screening)
- Presence of any other skin condition which may affect the evaluations of the study disease.
- Clinical picture suspicious for lichenoid drug eruption.
- Subjects whose lichen planus is predominantly hypertrophic, follicular, atrophic, or bullous variant.
- Lichen planus involving only mucosa or nails.
Trial locations and contacts
1 site in 1 country.
United States1 site
Virginia Clinical Research Inc.
Norfolk, Virginia, 23507
Stefanie A Hirano, MD, 757-625-0151, shirano@gmail.com
Clare E Foss, MD, 757-625-0151, fossce@evms.edu
About this study
Official title: An Open-Label Pilot Study to Evaluate the Safety and Efficacy of Apremilast (CC-10004) in the Treatment of Moderate to Severe Lichen Planus
This study is designed to demonstrate to efficacy and safety of Apremilast 20mg oral administration twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis is that the subjects will achieve a significant clinical improvement in their skin disease according to a specialized physician grading scale.
Read the detailed description
This is a phase 2, single center, non-randomized, open label efficacy and safety study designed to characterize the response of Apremilast 20 mg oral administered twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis and ideal primary end point will be that subjects achieve significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment.
Many various therapies have been used to treat LP including topical and oral corticosteroids, retinoids, cyclosporine, griseofulvin, dapsone and phototherapy, but often with disappointing response.4 It is an inflammatory condition whose pathogenesis involves damage to basal keratinocytes by alloreactive T cells through the release proinflammatory cytokines, such as TNF-α and IFN-γ.1 Significantly elevated levels of such inflammatory mediators are present in tissue from LP lesions compared to normal controls.5 Based on these observations, the investigation of Apremilast, due to its ability to inhibit multiple inflammatory cytokines, for the treatment of moderate to severe LP is warranted.
The primary objective of this study is to evaluate the clinical efficacy of Apremilast in subjects with moderate to severe lichen planus after 12 weeks of treatment. Other objectives are to evaluate the safety and tolerability of Apremilast, effects on quality of life, and efficacy for mucosal disease if present.
- Conditions
- Lichen Planus
- Being tested
- Apremilast (CC-10004)
- Keywords
- Lichen Planus
- Medical subject headings
- Lichen Planus
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Apremilast (CC-10004)Drug
Also called N-{2-[1-(3-ethoxy-4-methoxyphenyl)-2-(methylsulfonyl)ethyl]-1,3-dioxoisoindolin-4-yl}acetamide (S enantiomer)
Apremilast 20 mg tablet PO administered BID over 12 weeks
| Study group | Role | What they receive |
|---|---|---|
| Apremilast | Experimental | Apremilast 20 mg PO administered BID over 12 weeks Receives: Drug: Apremilast (CC-10004) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of subjects achieving significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment.
Measured: 12 weeks
Secondary outcomes (7)
Proportion of subjects with mucosal involvement who achieve a significant clinical response in mucosal disease defined as physician global assessment of mucosal disease (PGAMD) of "complete resolution" or "marked improvement" after 12 weeks of treatment.
Measured: 12 weeks
Change in the subjects' target area lesion count after 12 weeks of treatment relative to baseline.
Measured: 12 weeks
Change in the subjects' target area lesion severity score (TALSS) after 12 weeks of treatment relative to baseline.
Measured: 12 weeks
Proportion of subjects who achieve a subject global assessment of "complete resolution" or "marked improvement" after 12 weeks of treatment.
Measured: 12 weeks
Change in the subjects' dermatology life quality index (DLQI) score after 12 weeks of treatment relative to baseline.
Measured: 12 weeks
Change in the subjects' assessment of itching on a visual analogue scale (VAS) after 12 weeks of treatment relative to baseline.
Measured: 12 weeks
Safety and tolerability of Apremilast (type, frequency, severity, and relationship of adverse events to study treatment)
Measured: 16 weeks total (12 weeks treatment, 4 weeks observation)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jan 2010 (estimated)
- Study startFeb 2010
- Primary completionFeb 2012 (estimated)
- Study completionFeb 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Lichen PlanusStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Virginia Clinical Research, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Celgene Corporation
- Investigators
- David M Pariser, MD, Principal investigator, Virginia Clinical Research, Inc.Clare E Foss, MD, Study chair, Eastern Virginia Medical School
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01041625
- Sponsor's protocol ID
- VCR 001
- Other IDs
- 107047
- Status last verified
- Dec 2009
- First submitted
- 30 Dec 2009
- Last updated
- 1 Jan 2010
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Boyd AS, Neldner KH. Lichen planus. J Am Acad Dermatol. 1991 Oct;25(4):593-619. doi: 10.1016/0190-9622(91)70241-s.
PubMed 1791218Silverman S Jr, Gorsky M, Lozada-Nur F, Giannotti K. A prospective study of findings and management in 214 patients with oral lichen planus. Oral Surg Oral Med Oral Pathol. 1991 Dec;72(6):665-70. doi: 10.1016/0030-4220(91)90007-y.
PubMed 1812447Cribier B, Frances C, Chosidow O. Treatment of lichen planus. An evidence-based medicine analysis of efficacy. Arch Dermatol. 1998 Dec;134(12):1521-30. doi: 10.1001/archderm.134.12.1521.
PubMed 9875189Chen X, Liu Z, Yue Q. The expression of TNF-alpha and ICAM-1 in lesions of lichen planus and its implication. J Huazhong Univ Sci Technolog Med Sci. 2007 Dec;27(6):739-41. doi: 10.1007/s11596-007-0632-x.
PubMed 18231758Bolognia JL, Jorizzo JL, Rapini RP. Dermatology. 2nd ed. Philadelphia, Pa: Elsevier: 2008.
Paul J, Foss CE, Hirano SA, Cunningham TD, Pariser DM. An open-label pilot study of apremilast for the treatment of moderate to severe lichen planus: a case series. J Am Acad Dermatol. 2013 Feb;68(2):255-61. doi: 10.1016/j.jaad.2012.07.014. Epub 2012 Aug 19.
PubMed 22910104
Study at a glance
From the registry record, last updated by the sponsor on 1 Jan 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Pilot Study to Evaluate the Safety and Efficacy of Apremilast (CC-10004) in the Treatment of Moderate to Severe Lichen Planus
- ClinicalTrials.gov ID
- NCT01041625
- Acronym / sponsor ID
- VCR 001
- Conditions
- Lichen Planus
- Keywords
- Lichen Planus
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- Virginia Clinical Research, Inc.University, hospital or other
- Collaborators
- Celgene Corporation
- Study start
- Feb 2010
- Primary completion
- Feb 2012 (estimated)
- Study completion
- Feb 2012 (estimated)
- First posted
- 1 Jan 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is designed to demonstrate to efficacy and safety of Apremilast 20mg oral administration twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis is that the subjects will achieve a significant clinical improvement in their skin disease according to a specialized physician grading scale.
Detailed description
This is a phase 2, single center, non-randomized, open label efficacy and safety study designed to characterize the response of Apremilast 20 mg oral administered twice daily over 12 weeks in subjects with moderate to severe lichen planus. The hypothesis and ideal primary end point will be that subjects achieve significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment.
Many various therapies have been used to treat LP including topical and oral corticosteroids, retinoids, cyclosporine, griseofulvin, dapsone and phototherapy, but often with disappointing response.4 It is an inflammatory condition whose pathogenesis involves damage to basal keratinocytes by alloreactive T cells through the release proinflammatory cytokines, such as TNF-α and IFN-γ.1 Significantly elevated levels of such inflammatory mediators are present in tissue from LP lesions compared to normal controls.5 Based on these observations, the investigation of Apremilast, due to its ability to inhibit multiple inflammatory cytokines, for the treatment of moderate to severe LP is warranted.
The primary objective of this study is to evaluate the clinical efficacy of Apremilast in subjects with moderate to severe lichen planus after 12 weeks of treatment. Other objectives are to evaluate the safety and tolerability of Apremilast, effects on quality of life, and efficacy for mucosal disease if present.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Apremilast | Experimental | Drug: Apremilast (CC-10004)Apremilast 20 mg PO administered BID over 12 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- Must understand and voluntarily sign an informed consent form
- Must be male or female and aged ≥ 18 years at time of consent
- Must be able to adhere to the study visit schedule and other protocol requirements
- Subjects must have stable cutaneous lichen planus appropriate for systemic therapy based on the following criteria:
- Subjects using topical corticosteroids must be tapered and must undergo a washout period prior to initiation of the study. The washout period for topical corticosteroids is 2 weeks.
Key exclusion criteria (29 listed)
- Inability to provide voluntary consent
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Pregnant or breastfeeding
- Systemic fungal infection
- History of active mycobacterial infection with any species (including Mycobacterium tuberculosis) within 3 years prior to screening visit. Subjects with Mycobacterium tuberculosis infection more than 3 years prior to screening visit are allowed if successful treatment was completed at least 3 years prior to randomization and is documented and available for verification.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Apremilast (CC-10004)Also: N-{2-[1-(3-ethoxy-4-methoxyphenyl)-2-(methylsulfonyl)ethyl]-1,3-dioxoisoindolin-4-yl}acetamide (S enantiomer)Apremilast 20 mg tablet PO administered BID over 12 weeks | Drug | Apremilast |
Primary outcome
Proportion of subjects achieving significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment.
Time frame: 12 weeks
7 secondary outcomes
Proportion of subjects with mucosal involvement who achieve a significant clinical response in mucosal disease defined as physician global assessment of mucosal disease (PGAMD) of "complete resolution" or "marked improvement" after 12 weeks of treatment.
Time frame: 12 weeks
Change in the subjects' target area lesion count after 12 weeks of treatment relative to baseline.
Time frame: 12 weeks
Change in the subjects' target area lesion severity score (TALSS) after 12 weeks of treatment relative to baseline.
Time frame: 12 weeks
Proportion of subjects who achieve a subject global assessment of "complete resolution" or "marked improvement" after 12 weeks of treatment.
Time frame: 12 weeks
Change in the subjects' dermatology life quality index (DLQI) score after 12 weeks of treatment relative to baseline.
Time frame: 12 weeks
Change in the subjects' assessment of itching on a visual analogue scale (VAS) after 12 weeks of treatment relative to baseline.
Time frame: 12 weeks
Safety and tolerability of Apremilast (type, frequency, severity, and relationship of adverse events to study treatment)
Time frame: 16 weeks total (12 weeks treatment, 4 weeks observation)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Jan 2010.
- Background: PubMed 1791218Boyd AS, Neldner KH. Lichen planus. J Am Acad Dermatol. 1991 Oct;25(4):593-619. doi: 10.1016/0190-9622(91)70241-s.
- Background: PubMed 1812447Silverman S Jr, Gorsky M, Lozada-Nur F, Giannotti K. A prospective study of findings and management in 214 patients with oral lichen planus. Oral Surg Oral Med Oral Pathol. 1991 Dec;72(6):665-70. doi: 10.1016/0030-4220(91)90007-y.
- Background: PubMed 9875189Cribier B, Frances C, Chosidow O. Treatment of lichen planus. An evidence-based medicine analysis of efficacy. Arch Dermatol. 1998 Dec;134(12):1521-30. doi: 10.1001/archderm.134.12.1521.
- Background: PubMed 18231758Chen X, Liu Z, Yue Q. The expression of TNF-alpha and ICAM-1 in lesions of lichen planus and its implication. J Huazhong Univ Sci Technolog Med Sci. 2007 Dec;27(6):739-41. doi: 10.1007/s11596-007-0632-x.
- BackgroundBolognia JL, Jorizzo JL, Rapini RP. Dermatology. 2nd ed. Philadelphia, Pa: Elsevier: 2008.
- Derived from this study: PubMed 22910104Paul J, Foss CE, Hirano SA, Cunningham TD, Pariser DM. An open-label pilot study of apremilast for the treatment of moderate to severe lichen planus: a case series. J Am Acad Dermatol. 2013 Feb;68(2):255-61. doi: 10.1016/j.jaad.2012.07.014. Epub 2012 Aug 19.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
David M Pariser, MD principal investigator
Virginia Clinical Research, Inc.
Clare E Foss, MD study chair
Eastern Virginia Medical School
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Apremilast (CC-10004) in people with Lichen Planus, run by Virginia Clinical Research, Inc. at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 29 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Jan 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Stefanie A Hirano, MD (757-625-0151, shirano@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of subjects achieving significant clinical response in cutaneous disease defined as a 2 or more grade improvement of the physician global assessment (PGA) after 12 weeks of treatment. (time frame: 12 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01041625, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Jan 2010.